JP7412001B2 - 改変されたCas9タンパク質及びその用途 - Google Patents

改変されたCas9タンパク質及びその用途 Download PDF

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JP7412001B2
JP7412001B2 JP2020523211A JP2020523211A JP7412001B2 JP 7412001 B2 JP7412001 B2 JP 7412001B2 JP 2020523211 A JP2020523211 A JP 2020523211A JP 2020523211 A JP2020523211 A JP 2020523211A JP 7412001 B2 JP7412001 B2 JP 7412001B2
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protein
cells
dsacas9
amino acid
sequence
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JPWO2019235627A1 (ja
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哲也 山形
ユアンバウ シン
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Modalis Therapeutics Corp
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    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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JP2020523211A 2018-06-08 2019-06-07 改変されたCas9タンパク質及びその用途 Active JP7412001B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862682244P 2018-06-08 2018-06-08
US62/682,244 2018-06-08
PCT/JP2019/022795 WO2019235627A1 (ja) 2018-06-08 2019-06-07 改変されたCas9タンパク質及びその用途

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JPWO2019235627A1 JPWO2019235627A1 (ja) 2021-06-24
JP7412001B2 true JP7412001B2 (ja) 2024-01-12

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US (1) US20220017881A1 (de)
EP (1) EP3805386A4 (de)
JP (1) JP7412001B2 (de)
KR (1) KR20210025046A (de)
CN (1) CN112513266A (de)
AU (1) AU2019281158A1 (de)
BR (1) BR112020024992A2 (de)
CA (1) CA3103088A1 (de)
IL (1) IL279178A (de)
MX (1) MX2020013158A (de)
SG (1) SG11202012228QA (de)
WO (1) WO2019235627A1 (de)
ZA (1) ZA202100092B (de)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020283372A1 (en) 2019-05-28 2021-11-18 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting DMPK gene
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
WO2022045366A1 (en) * 2020-08-31 2022-03-03 Modalis Therapeutics Corporation Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene
WO2022114243A1 (en) 2020-11-25 2022-06-02 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008008523A1 (en) 2006-07-14 2008-01-17 Regents Of The University Of Minnesota COMPOUNDS THAT BIND α5β1 INTEGRIN AND METHODS OF USE
JP2010505437A (ja) 2006-10-13 2010-02-25 ノボ ノルディスク ヘルス ケア アーゲー 塩基性タンパク質タグに融合したプロセシング酵素
JP2016533332A (ja) 2013-09-24 2016-10-27 マサチューセッツ インスティテュート オブ テクノロジー 自己集合型ナノ粒子ワクチン
WO2016196655A1 (en) 2015-06-03 2016-12-08 The Regents Of The University Of California Cas9 variants and methods of use thereof
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
JP2017527284A (ja) 2014-09-01 2017-09-21 ブイアイビー ブイゼットダブリュVib Vzw 変異体ポア
WO2017217768A1 (ko) 2016-06-15 2017-12-21 주식회사 툴젠 온타겟 및 오프타겟의 다중 타겟 시스템을 이용하는, 표적 특이적 유전자 가위 스크리닝 방법 및 이의 용도
JP2018502572A5 (de) 2015-12-31 2019-02-14

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KR102670705B1 (ko) * 2015-01-02 2024-05-31 다케다 파머수티컬 컴패니 리미티드 혈장 칼리크레인 및 인자 xii에 대한 이중특이성 항체
EP3265559B1 (de) 2015-03-03 2021-01-06 The General Hospital Corporation Manipulierte crispr-cas9-nukleasen mit veränderter pam-spezifität
JP7107683B2 (ja) * 2015-06-18 2022-07-27 ザ・ブロード・インスティテュート・インコーポレイテッド オフターゲット効果を低下させるcrispr酵素突然変異
JPWO2017010543A1 (ja) 2015-07-14 2018-06-14 国立大学法人 東京大学 改変されたFnCas9タンパク質及びその使用
SG10201913505WA (en) 2016-10-17 2020-02-27 Univ Nanyang Tech Truncated crispr-cas proteins for dna targeting
CN110662835B (zh) 2017-05-19 2023-04-28 清华大学 工程化改造用于由增强的指导RNA优化的基因编辑和转录调节的最小化SaCas9 CRISPR/Cas系统
WO2019089910A1 (en) * 2017-11-01 2019-05-09 Ohio State Innovation Foundation Highly compact cas9-based transcriptional regulators for in vivo gene regulation
BR112021009481A2 (pt) * 2018-11-16 2021-08-17 Astellas Pharma Inc. método para tratamento da distrofia muscular direcionando gene de utrofina.

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008008523A1 (en) 2006-07-14 2008-01-17 Regents Of The University Of Minnesota COMPOUNDS THAT BIND α5β1 INTEGRIN AND METHODS OF USE
JP2010505437A (ja) 2006-10-13 2010-02-25 ノボ ノルディスク ヘルス ケア アーゲー 塩基性タンパク質タグに融合したプロセシング酵素
JP2016533332A (ja) 2013-09-24 2016-10-27 マサチューセッツ インスティテュート オブ テクノロジー 自己集合型ナノ粒子ワクチン
JP2017527284A (ja) 2014-09-01 2017-09-21 ブイアイビー ブイゼットダブリュVib Vzw 変異体ポア
WO2016196655A1 (en) 2015-06-03 2016-12-08 The Regents Of The University Of California Cas9 variants and methods of use thereof
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
JP2018502572A5 (de) 2015-12-31 2019-02-14
WO2017217768A1 (ko) 2016-06-15 2017-12-21 주식회사 툴젠 온타겟 및 오프타겟의 다중 타겟 시스템을 이용하는, 표적 특이적 유전자 가위 스크리닝 방법 및 이의 용도

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Title
MA, Dacheng et al.,Rational Design of Mini-Cas9 for Transcriptional Activation,ACS Synth. Biol.,2018年03月21日,Vol. 7,pp. 978-985, Supporting Information
NISHIMASU, Hiroshi et al.,Crystal structure of staphylococcus aureus Cas9,Cell,2015年,Vol. 162,pp. 1113-1126, Figure S3

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IL279178A (en) 2021-01-31
CN112513266A (zh) 2021-03-16
CA3103088A1 (en) 2019-12-12
KR20210025046A (ko) 2021-03-08
WO2019235627A1 (ja) 2019-12-12
US20220017881A1 (en) 2022-01-20
MX2020013158A (es) 2021-04-29
BR112020024992A2 (pt) 2021-03-23
EP3805386A4 (de) 2022-03-23
JPWO2019235627A1 (ja) 2021-06-24
EP3805386A1 (de) 2021-04-14
AU2019281158A1 (en) 2021-01-14
ZA202100092B (en) 2021-10-27
SG11202012228QA (en) 2021-01-28

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